Pemafibrate

Summary

Pemafibrate (CHEMBL247951) is an approved small-molecule PPARα agonist (ATC C10AB12); indicated across 2 conditions including cardiovascular disorder and diabetic retinopathy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: C10AB12
• Indications: 2 conditions
• Clinical trials: 2
• Chemistry: 490.5 Da · C28H30N2O6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL247951
Name	Pemafibrate
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	11526038
ChEBI	CHEBI:228365
ATC	C10AB12
Molecular formula	C28H30N2O6
Molecular weight	490.5
InChIKey	ZHKNLJLMDFQVHJ-RUZDIDTESA-N

SMILES: CC[C@H](C(=O)O)OC1=CC=CC(=C1)CN(CCCOC2=CC=C(C=C2)OC)C3=NC4=CC=CC=C4O3

IUPAC name: (2R)-2-[3-[[1,3-benzoxazol-2-yl-[3-(4-methoxyphenoxy)propyl]amino]methyl]phenoxy]butanoic acid

ChEBI definition: A member of the class of 1,3-benzoxazoles that is 1,3-benzoxazol-2-amine in which the amino hydrogens are replaced by 3-[(1R)-1-carboxypropoxy]benzyl and 3-(4-methoxyphenoxy)propyl groups. It is a selective peroxisome proliferator-activated receptor (PPAR)-α agonist that is used for the treatment of hyperlipidaemia.

Pharmacological roles (ChEBI): PPARα agonist, antilipemic drug, hepatoprotective agent.

Also known as: (R)-K-13675, K-13675, (r)-, Pemafibrate, Pemafibrato, PEMAFIBRATE

Patent coverage: 217 distinct patent families (604 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 601 (100%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Peroxisome proliferator-activated receptor gamma, Peroxisome proliferator-activated receptor alpha, Peroxisome proliferator-activated receptor delta.

Bioactivity

ChEMBL activities: 7 potent at pChembl ≥ 5 of 7 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
PPARA	9	EC50	1	nM	CHEMBL_ACT_14985376
PPARA	9	EC50	1	nM	CHEMBL_ACT_2035022
PPARA	9	EC50	1	nM	CHEMBL_ACT_25110708
PPARG	5.96	EC50	1100	nM	CHEMBL_ACT_2035055
PPARD	5.8	EC50	1580	nM	CHEMBL_ACT_2035092
PPARG	5.77	EC50	1700	nM	CHEMBL_ACT_14985386
PPARD	5	EC50	10000	nM	CHEMBL_ACT_14985381

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
cardiovascular disorder	4	MONDO:0004995	EFO:0000319

1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
diabetic retinopathy	3	MONDO:0005266	EFO:0003770

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE3	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03345901	PHASE3	TERMINATED	PROMINENT-Eye Ancillary Study (Protocol AD)
NCT07562178	Not specified	RECRUITING	Pemafibrate for Symptomatic ICAS RCT

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).